Trial Outcomes & Findings for A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (NCT NCT03191799)

NCT ID: NCT03191799

Last Updated: 2021-06-11

Results Overview

Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. aPCC = activated prothrombin complex concentrate

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

195 participants

Primary outcome timeframe

From Baseline until study completion (up to 2 years)

Results posted on

2021-06-11

Participant Flow

A total of 195 patients were enrolled in the clinical trial.

Participant milestones

Participant milestones
Measure	1.5 mg/kg Emicizumab QW Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Study STARTED	195
Overall Study Received at Least One Dose of Emicizumab	193
Overall Study Dose Up-Titrated to 3 mg/kg QW	2
Overall Study COMPLETED	186
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	1.5 mg/kg Emicizumab QW Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Study Withdrawal by Subject	4
Overall Study Physician Decision	2
Overall Study Lost to Follow-up	1
Overall Study Death	2

Baseline Characteristics

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Age, Continuous	32.2 Years STANDARD_DEVIATION 16.6 • n=5 Participants
Age, Customized ≥12 to <18 Years Old	40 Participants n=5 Participants
Age, Customized ≥18 to <65 Years Old	146 Participants n=5 Participants
Age, Customized ≥65 Years Old	9 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	195 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	41 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	143 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	11 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	19 Participants n=5 Participants
Race (NIH/OMB) Asian	38 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	7 Participants n=5 Participants
Race (NIH/OMB) White	121 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline until study completion (up to 2 years)

Population: Safety Population: all participants who received at least one dose of emicizumab

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Any Adverse Event (AE)	163 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Fatal AE	2 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Serious AE	31 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale AE Leading to Withdrawal from Treatment	1 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale AE Leading to Dose Modification/Interruption	4 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale AE Leading to Study Discontinuation	1 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Grade 3-5 AE	39 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Related AE	35 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Local Injection Site Reaction	22 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Systemic Hypersensitivity/Anaphylac(tic/toid) Reaction	0 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Thromboembolic Event (TE)	2 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale TE Related to aPCC and Emicizumab	0 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Thrombotic Microangiopathy (TMA)	0 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale TMA Related to aPCC and Emicizumab	0 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Cases of Potential Drug-Induced Liver Injury	0 Participants
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale Suspected Transmission of Infectious Agent by the Study Drug	0 Participants

PRIMARY outcome

Timeframe: From Baseline until study completion (up to 2 years)

Population: Safety Population: all participants who received at least one dose of emicizumab

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=800 Adverse Events (AEs) Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs All-Grade AEs	211.92 AEs per 100 patient-years Interval 197.49 to 227.13
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs Serious AEs	13.25 AEs per 100 patient-years Interval 9.83 to 17.46
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs Grade ≥3 AEs	17.22 AEs per 100 patient-years Interval 13.29 to 21.95

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Population: Safety Population: all participants who received at least one dose of emicizumab

The World Health Organization (WHO) toxicity grading scale was used for determining the severity of laboratory abnormalities (i.e., test results outside of the reference range) for hematology and biochemistry parameters; Grade 0 is normal and Grades 1 to 4 represent worsening levels of the parameter outside of the normal range in the specified direction of the abnormality (high and low are above and below the range, respectively). Not every laboratory abnormality qualified as an adverse event (AE). A laboratory test result was reported as an AE if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment; resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. Baseline was defined as the last available assessment prior to first receipt of study drug. Abs = absolute count; SGOT/AST = aspartate aminotransferase; SGPT/ALT = alanine aminotransferase

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 0 to 0	173 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 0 to 1	8 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 1 to 0	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 1 to 3	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 2 to 3	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 0 to 0	173 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 0 to 1	13 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 0 to 4	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Platelets, Low - Grade 0 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Alkaline Phosphatase, High - Grade 0 to 0	187 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Alkaline Phosphatase, High - Grade 0 to 1	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 0 to 0	169 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 1 to 0	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Blood Urea Nitrogen, High - Grade 0 to 0	187 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), Low - Grade 0 to 0	173 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), Low - Grade 0 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), Low - Grade 1 to 1	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Creatinine, High - Grade 0 to 1	5 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Creatinine, High - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 0 to 1	28 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 0 to 2	6 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 1 to 2	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 2 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 2 to 3	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 3 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 3 to 3	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 4 to 3	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Magnesium, Low - Grade 1 to 1	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Phosphorus, Low - Grade 0 to 0	160 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Phosphorus, Low - Grade 0 to 1	21 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Phosphorus, Low - Grade 1 to 0	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Phosphorus, Low - Grade 1 to 1	9 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Phosphorus, Low - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, High - Grade 0 to 0	189 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, High - Grade 0 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, High - Grade 0 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, Low - Grade 0 to 0	184 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, Low - Grade 0 to 1	6 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, Low - Grade 1 to 0	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 0 to 0	164 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 0 to 0	158 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 0 to 1	22 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 1 to 1	4 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 1 to 2	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, High - Grade 0 to 0	178 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, High - Grade 0 to 1	7 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 0 to 2	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 1 to 0	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 0 to 2	4 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 0 to 4	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Hemoglobin, Low - Grade 2 to 1	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 0 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 0 to 3	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Neutrophils, Total (Abs), Low - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Platelets, Low - Grade 0 to 0	190 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, Low - Grade 0 to 0	180 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, Low - Grade 0 to 1	7 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, Low - Grade 0 to 2	4 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, Low - Grade 1 to 0	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Magnesium, Low - Grade 0 to 0	188 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Magnesium, Low - Grade 0 to 1	4 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Platelets, Low - Grade 1 to 0	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Alkaline Phosphatase, High - Grade 0 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Alkaline Phosphatase, High - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 0 to 1	14 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 1 to 2	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Bilirubin, High - Grade 2 to 2	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Blood Urea Nitrogen, High - Grade 0 to 1	5 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Blood Urea Nitrogen, High - Grade 1 to 1	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), High - Grade 0 to 0	192 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), High - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), Low - Grade 0 to 1	15 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Calcium (Corrected), Low - Grade 1 to 0	3 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Creatinine, High - Grade 0 to 0	185 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Creatinine, High - Grade 0 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Creatinine, High - Grade 1 to 1	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Glucose, High - Grade 0 to 0	148 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Potassium, High - Grade 0 to 4	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 0 to 1	13 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 0 to 2	8 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 1 to 0	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 1 to 1	5 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 1 to 2	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGOT/AST, High - Grade 1 to 3	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 0 to 2	5 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 1 to 0	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline SGPT/ALT, High - Grade 1 to 4	1 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, High - Grade 0 to 2	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, High - Grade 1 to 0	4 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, High - Grade 1 to 1	2 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 0 to 0	166 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 0 to 1	19 Participants
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline Sodium, Low - Grade 1 to 1	2 Participants

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Population: Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.

Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Body Temperature at Specified Timepoints Baseline (BL) - Value at Visit	36.33 degrees Celsius Standard Deviation 0.52
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Week 2	0.04 degrees Celsius Standard Deviation 0.41
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Week 3	0.03 degrees Celsius Standard Deviation 0.40
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Week 5	0.03 degrees Celsius Standard Deviation 0.45
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Month 3	0.04 degrees Celsius Standard Deviation 0.45
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Month 6	0.07 degrees Celsius Standard Deviation 0.50
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Month 9	0.06 degrees Celsius Standard Deviation 0.48
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Month 12	0.07 degrees Celsius Standard Deviation 0.48
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Month 18	0.10 degrees Celsius Standard Deviation 0.53
Change From Baseline in Body Temperature at Specified Timepoints Change from BL at Early Termination/Study Completion	0.08 degrees Celsius Standard Deviation 0.48

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Month 3	0.1 millimetres of mercury (mmHg) Standard Deviation 11.5
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Month 6	-0.2 millimetres of mercury (mmHg) Standard Deviation 13.1
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Month 9	0.1 millimetres of mercury (mmHg) Standard Deviation 13.1
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Month 12	0.1 millimetres of mercury (mmHg) Standard Deviation 13.9
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Early Termination/Study Completion	1.9 millimetres of mercury (mmHg) Standard Deviation 14.1
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Baseline (BL) - Value at Visit	121.1 millimetres of mercury (mmHg) Standard Deviation 14.3
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Week 2	-0.9 millimetres of mercury (mmHg) Standard Deviation 11.1
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Week 3	-1.2 millimetres of mercury (mmHg) Standard Deviation 10.6
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Week 5	-1.3 millimetres of mercury (mmHg) Standard Deviation 12.3
Change From Baseline in Systolic Blood Pressure at Specified Timepoints Change from BL at Month 18	-0.1 millimetres of mercury (mmHg) Standard Deviation 13.8

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Baseline (BL) - Value at Visit	75.5 millimetres of mercury (mmHg) Standard Deviation 10.4
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Week 2	-1.2 millimetres of mercury (mmHg) Standard Deviation 8.5
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Week 3	-1.0 millimetres of mercury (mmHg) Standard Deviation 8.9
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Week 5	-1.2 millimetres of mercury (mmHg) Standard Deviation 9.2
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Month 3	-0.3 millimetres of mercury (mmHg) Standard Deviation 9.8
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Month 6	-0.7 millimetres of mercury (mmHg) Standard Deviation 10.1
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Month 9	-1.3 millimetres of mercury (mmHg) Standard Deviation 9.4
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Month 12	-1.2 millimetres of mercury (mmHg) Standard Deviation 10.4
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Month 18	-0.9 millimetres of mercury (mmHg) Standard Deviation 9.6
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints Change from BL at Early Termination/Study Completion	0.0 millimetres of mercury (mmHg) Standard Deviation 11.0

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Pulse Rate at Specified Timepoints Baseline (BL) - Value at Visit	76.6 beats per minute Standard Deviation 11.6
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Week 2	1.0 beats per minute Standard Deviation 9.9
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Week 3	1.6 beats per minute Standard Deviation 11.6
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Week 5	-0.5 beats per minute Standard Deviation 11.3
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Month 3	-0.8 beats per minute Standard Deviation 10.4
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Month 6	-0.7 beats per minute Standard Deviation 11.6
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Month 9	-1.0 beats per minute Standard Deviation 11.3
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Month 12	-0.5 beats per minute Standard Deviation 12.2
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Month 18	-2.1 beats per minute Standard Deviation 12.7
Change From Baseline in Pulse Rate at Specified Timepoints Change from BL at Early Termination/Study Completion	-1.2 beats per minute Standard Deviation 11.9

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Respiratory Rate at Specified Timepoints Baseline (BL) - Value at Visit	17.0 breaths per minute Standard Deviation 3.1
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Week 2	0.2 breaths per minute Standard Deviation 2.2
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Week 3	0.0 breaths per minute Standard Deviation 2.3
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Week 5	-0.1 breaths per minute Standard Deviation 2.5
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Month 3	0.3 breaths per minute Standard Deviation 4.7
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Month 6	-0.1 breaths per minute Standard Deviation 2.5
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Month 9	0.0 breaths per minute Standard Deviation 2.7
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Month 12	0.3 breaths per minute Standard Deviation 2.9
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Month 18	-0.4 breaths per minute Standard Deviation 2.7
Change From Baseline in Respiratory Rate at Specified Timepoints Change from BL at Early Termination/Study Completion	-0.7 breaths per minute Standard Deviation 3.1

PRIMARY outcome

Timeframe: Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Week 2	0.14 kilograms (kg) Standard Deviation 1.06
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Week 3	0.34 kilograms (kg) Standard Deviation 1.18
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Week 5	0.52 kilograms (kg) Standard Deviation 1.56
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Month 3	0.98 kilograms (kg) Standard Deviation 2.44
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Month 6	1.11 kilograms (kg) Standard Deviation 3.20
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Month 9	1.61 kilograms (kg) Standard Deviation 4.08
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Month 12	1.85 kilograms (kg) Standard Deviation 4.88
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Early Termination/Study Completion	3.15 kilograms (kg) Standard Deviation 6.78
Change From Baseline in Body Weight at Specified Timepoints Baseline (BL) - Value at Visit	69.55 kilograms (kg) Standard Deviation 16.60
Change From Baseline in Body Weight at Specified Timepoints Change from BL at Month 18	2.58 kilograms (kg) Standard Deviation 5.56

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

The number of bleeds over the efficacy period was analyzed as an ABR using a negative binomial regression model. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all bleed types, bleeds due to surgery/procedure were excluded. Bleeds occurring after dose up-titration have been excluded.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Bleeds	0.5 bleeds per year Interval 0.27 to 0.89
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds All Bleeds	1.1 bleeds per year Interval 0.8 to 1.47
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Joint Bleeds	0.4 bleeds per year Interval 0.15 to 0.86
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Target Joint Bleeds	0.2 bleeds per year Interval 0.07 to 0.68
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Spontaneous Bleeds	0.3 bleeds per year Interval 0.15 to 0.73

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

The number of bleeds over the efficacy period was calculated as: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all types, bleeds due to surgery/procedure and bleeds occurring after dose up-titration were excluded.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Bleeds	0.6 bleeds per year Interval 0.0 to 4.85
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds All Bleeds	1.3 bleeds per year Interval 0.06 to 6.02
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Joint Bleeds	0.4 bleeds per year Interval 0.0 to 4.55
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Target Joint Bleeds	0.3 bleeds per year Interval 0.0 to 4.28
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Spontaneous Bleeds	0.4 bleeds per year Interval 0.0 to 4.49

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Bleeds	0.0 bleeds per year Interval 0.0 to 0.0
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Joint Bleeds	0.0 bleeds per year Interval 0.0 to 0.0
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Target Joint Bleeds	0.0 bleeds per year Interval 0.0 to 0.0
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds Treated Spontaneous Bleeds	0.0 bleeds per year Interval 0.0 to 0.0
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds All Bleeds	0.0 bleeds per year Interval 0.0 to 1.01

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds 0 Bleeds	82.6 Percentage of participants Interval 76.5 to 87.6
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds 1-3 Bleeds	12.3 Percentage of participants Interval 8.0 to 17.8
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds 4-10 Bleeds	4.1 Percentage of participants Interval 1.8 to 7.9
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds >10 Bleeds	1.0 Percentage of participants Interval 0.1 to 3.7

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds 0 Bleeds	54.9 Percentage of participants Interval 47.6 to 62.0
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds 1-3 Bleeds	30.3 Percentage of participants Interval 23.9 to 37.2
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds 4-10 Bleeds	12.3 Percentage of participants Interval 8.0 to 17.8
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds >10 Bleeds	2.6 Percentage of participants Interval 0.8 to 5.9

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds >10 Bleeds	0.5 Percentage of participants Interval 0.0 to 2.8
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds 0 Bleeds	89.2 Percentage of participants Interval 84.0 to 93.2
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds 1-3 Bleeds	8.7 Percentage of participants Interval 5.2 to 13.6
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds 4-10 Bleeds	1.5 Percentage of participants Interval 0.3 to 4.4

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds ABR 1-3.4	6.2 Percentage of participants Interval 3.2 to 10.5
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds ABR 3.5-10	2.6 Percentage of participants Interval 0.8 to 5.9
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds ABR <1	90.3 Percentage of participants Interval 85.2 to 94.0
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds ABR >10	1.0 Percentage of participants Interval 0.1 to 3.7

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds ABR >10	2.6 Percentage of participants Interval 0.8 to 5.9
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds ABR <1	72.3 Percentage of participants Interval 65.5 to 78.5
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds ABR 1-3.4	20.5 Percentage of participants Interval 15.1 to 26.9
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds ABR 3.5-10	4.6 Percentage of participants Interval 2.1 to 8.6

SECONDARY outcome

Timeframe: From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)

Population: Intent-to-Treat (ITT) Population: all enrolled participants

The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds ABR <1	95.4 Percentage of participants Interval 91.4 to 97.9
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds ABR 1-3.4	2.6 Percentage of participants Interval 0.8 to 5.9
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds ABR 3.5-10	1.0 Percentage of participants Interval 0.1 to 3.7
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds ABR >10	1.0 Percentage of participants Interval 0.1 to 3.7

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. The Total Score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-17.44 score on a scale Standard Deviation 14.16
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	41.09 score on a scale Standard Deviation 16.15
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-17.39 score on a scale Standard Deviation 12.86
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-16.40 score on a scale Standard Deviation 14.58
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-16.27 score on a scale Standard Deviation 15.39
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-14.13 score on a scale Standard Deviation 13.70

SECONDARY outcome

Timeframe: At 3, 6, 12, and 18 months

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a post-baseline value at a given timepoint.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints Month 6	72.4 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints Month 12	70.8 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints Month 3	78.2 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints Month 18	69.9 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	51.13 score on a scale Standard Deviation 23.02
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-27.36 score on a scale Standard Deviation 20.70
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-25.21 score on a scale Standard Deviation 23.31
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-23.29 score on a scale Standard Deviation 25.16
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-26.10 score on a scale Standard Deviation 23.12
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-23.14 score on a scale Standard Deviation 24.51

SECONDARY outcome

Timeframe: At 3, 6, 12, and 18 months

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life. An improvement larger than the responder threshold was defined as a decrease from baseline of at least 10 points in the Haem-A-QoL Physical Health domain score.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints Month 3	79.6 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints Month 6	74.5 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints Month 12	72.9 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints Month 18	71.3 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	39.40 score on a scale Standard Deviation 19.89
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-23.44 score on a scale Standard Deviation 22.07
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-21.01 score on a scale Standard Deviation 23.74
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-22.21 score on a scale Standard Deviation 22.57
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-20.34 score on a scale Standard Deviation 25.37
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-21.16 score on a scale Standard Deviation 22.31

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-21.43 score on a scale Standard Deviation 26.65
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-18.83 score on a scale Standard Deviation 29.54
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	39.23 score on a scale Standard Deviation 25.31
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-21.40 score on a scale Standard Deviation 26.51
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-21.91 score on a scale Standard Deviation 27.17
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-22.43 score on a scale Standard Deviation 25.55

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	26.61 score on a scale Standard Deviation 19.76
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	5.32 score on a scale Standard Deviation 30.06
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	5.73 score on a scale Standard Deviation 28.14
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	5.24 score on a scale Standard Deviation 29.18
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	7.70 score on a scale Standard Deviation 29.75
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	10.60 score on a scale Standard Deviation 33.42

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-19.55 score on a scale Standard Deviation 25.43
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	36.25 score on a scale Standard Deviation 24.53
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-21.22 score on a scale Standard Deviation 22.69
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-20.88 score on a scale Standard Deviation 24.11
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-22.37 score on a scale Standard Deviation 23.78
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-17.95 score on a scale Standard Deviation 23.62

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-8.64 score on a scale Standard Deviation 20.95
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-9.07 score on a scale Standard Deviation 20.78
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-10.83 score on a scale Standard Deviation 22.51
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-9.38 score on a scale Standard Deviation 24.81
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	25.64 score on a scale Standard Deviation 28.49
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-12.39 score on a scale Standard Deviation 25.20

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	42.70 score on a scale Standard Deviation 21.95
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-14.43 score on a scale Standard Deviation 19.83
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-13.29 score on a scale Standard Deviation 19.56
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-15.56 score on a scale Standard Deviation 17.63
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-15.46 score on a scale Standard Deviation 17.83
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-14.46 score on a scale Standard Deviation 20.10

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-9.72 score on a scale Standard Deviation 28.16
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	23.17 score on a scale Standard Deviation 28.65
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-7.35 score on a scale Standard Deviation 25.25
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-6.03 score on a scale Standard Deviation 22.76
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-7.74 score on a scale Standard Deviation 27.60
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-4.05 score on a scale Standard Deviation 21.32

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	68.86 score on a scale Standard Deviation 21.24
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-19.65 score on a scale Standard Deviation 23.67
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-17.63 score on a scale Standard Deviation 25.86
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-22.56 score on a scale Standard Deviation 22.17
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-20.20 score on a scale Standard Deviation 25.86
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-20.65 score on a scale Standard Deviation 28.65

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=155 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Baseline (BL) - Value at Visit	43.57 score on a scale Standard Deviation 22.05
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 3	-13.51 score on a scale Standard Deviation 20.63
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 6	-15.04 score on a scale Standard Deviation 21.19
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 12	-15.71 score on a scale Standard Deviation 19.88
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Change from BL at Month 18	-15.19 score on a scale Standard Deviation 21.88
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants Change from BL at Early Termination/Study Completion	-9.50 score on a scale Standard Deviation 20.70

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. The Total Score is derived from the scores for all domains and scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	35.10 score on a scale Standard Deviation 12.42
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-9.60 score on a scale Standard Deviation 12.51
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-12.65 score on a scale Standard Deviation 12.72
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-13.57 score on a scale Standard Deviation 13.20
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-14.02 score on a scale Standard Deviation 11.83
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-18.37 score on a scale Standard Deviation 17.53

SECONDARY outcome

Timeframe: At 3, 6, 12, and 18 months

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a post-baseline value at a given timepoint.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints Month 18	69.2 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints Month 3	63.9 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints Month 6	75.7 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints Month 12	75.0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-29.78 score on a scale Standard Deviation 17.55
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-30.71 score on a scale Standard Deviation 18.83
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	42.14 score on a scale Standard Deviation 18.40
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-29.10 score on a scale Standard Deviation 20.12
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-30.47 score on a scale Standard Deviation 20.74
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-34.03 score on a scale Standard Deviation 25.66

SECONDARY outcome

Timeframe: At 3, 6, 12, and 18 months

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life. An improvement larger than the responder threshold was defined as a decrease of at least 10 points in the Haemo-QoL-SF Physical Health domain score.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints Month 12	75.0 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints Month 3	77.8 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints Month 6	78.4 Percentage of participants
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints Month 18	79.5 Percentage of participants

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-8.64 score on a scale Standard Deviation 23.23
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-10.74 score on a scale Standard Deviation 21.31
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-12.68 score on a scale Standard Deviation 21.68
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-20.83 score on a scale Standard Deviation 23.87
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	31.61 score on a scale Standard Deviation 18.12
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-9.38 score on a scale Standard Deviation 20.94

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	62.32 score on a scale Standard Deviation 21.30
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-15.82 score on a scale Standard Deviation 25.55
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-20.04 score on a scale Standard Deviation 29.27
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-22.07 score on a scale Standard Deviation 26.71
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-28.21 score on a scale Standard Deviation 27.80
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-23.61 score on a scale Standard Deviation 28.16

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	24.11 score on a scale Standard Deviation 14.79
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-7.23 score on a scale Standard Deviation 18.25
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-7.72 score on a scale Standard Deviation 17.75
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-10.35 score on a scale Standard Deviation 20.24
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-3.39 score on a scale Standard Deviation 25.70
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-12.50 score on a scale Standard Deviation 23.48

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-16.36 score on a scale Standard Deviation 21.54
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	26.43 score on a scale Standard Deviation 25.10
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-9.57 score on a scale Standard Deviation 20.08
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-13.09 score on a scale Standard Deviation 22.80
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-15.18 score on a scale Standard Deviation 25.51
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-23.96 score on a scale Standard Deviation 32.67

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	46.07 score on a scale Standard Deviation 20.79
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-6.84 score on a scale Standard Deviation 15.66
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-11.95 score on a scale Standard Deviation 20.84
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-14.84 score on a scale Standard Deviation 23.32
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-15.36 score on a scale Standard Deviation 21.67
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-15.28 score on a scale Standard Deviation 19.08

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	35.95 score on a scale Standard Deviation 26.02
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-0.52 score on a scale Standard Deviation 24.95
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-3.19 score on a scale Standard Deviation 28.13
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-5.47 score on a scale Standard Deviation 27.57
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-5.95 score on a scale Standard Deviation 36.41
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-7.41 score on a scale Standard Deviation 31.56

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-8.01 score on a scale Standard Deviation 18.53
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	21.43 score on a scale Standard Deviation 17.50
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-2.93 score on a scale Standard Deviation 18.72
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-5.51 score on a scale Standard Deviation 19.08
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-6.79 score on a scale Standard Deviation 17.51
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-11.81 score on a scale Standard Deviation 18.55

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=40 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Baseline (BL) - Value at Visit	26.07 score on a scale Standard Deviation 22.04
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 3	-2.73 score on a scale Standard Deviation 26.03
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 6	-8.27 score on a scale Standard Deviation 23.27
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 12	-5.08 score on a scale Standard Deviation 24.82
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Month 18	-5.89 score on a scale Standard Deviation 21.54
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants Change from BL at Early Termination/Study Completion	-13.19 score on a scale Standard Deviation 26.59

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The index utility score is based on the participant's assessment of their health on five scales: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. It ranges from -0.224 to 1, with 0 corresponding to death and 1 corresponding to full health; negative values correspond to health states worse than death. Higher scores indicate better quality of life.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Baseline (BL) - Value at Visit	0.67 score on a scale Standard Deviation 0.23
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Change from BL at Month 3	0.08 score on a scale Standard Deviation 0.18
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Change from BL at Month 6	0.11 score on a scale Standard Deviation 0.18
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Change from BL at Month 12	0.09 score on a scale Standard Deviation 0.19
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Change from BL at Month 18	0.07 score on a scale Standard Deviation 0.21
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints Change from BL at Early Termination/Study Completion	0.10 score on a scale Standard Deviation 0.22

SECONDARY outcome

Timeframe: Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)

Population: The number analyzed represents the number of participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.

The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The visual analogue scale (VAS) ranges from 0 to 100 points, on which the participant self-assesses their current health status; 0 being the worst health you can imagine and 100 being the best health you can imagine. Higher scores are reflective of better health.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=195 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Change from BL at Month 3	8.02 score on a scale Standard Deviation 18.70
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Baseline (BL) - Value at Visit	71.01 score on a scale Standard Deviation 19.41
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Change from BL at Month 6	10.27 score on a scale Standard Deviation 17.24
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Change from BL at Month 12	10.69 score on a scale Standard Deviation 18.18
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Change from BL at Month 18	9.49 score on a scale Standard Deviation 21.08
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints Change from BL at Early Termination/Study Completion	14.52 score on a scale Standard Deviation 17.91

SECONDARY outcome

Timeframe: Month 3

Population: The number of participants analyzed represents the total number who responded to this questionnaire.

The EmiPref questionnaire asked participants to specify the treatment they would prefer to continue to receive after receiving treatment with their previous episodic or prophylactic regimen and subcutaneous (SC) emicizumab, or if they had no preference. The 95% confidence intervals were calculated for one sample binomial using the Pearson-Clopper method.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=179 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire Prefer the New Study Drug Treatment (Emicizumab)	96.6 Percentage of participants Interval 92.85 to 98.76
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire Prefer my Old Hemophilia Treatment	0.6 Percentage of participants Interval 0.01 to 3.07
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire Have no Preference	2.8 Percentage of participants Interval 0.91 to 6.4

SECONDARY outcome

Timeframe: Baseline (Week 1), Week 5, and at 3, 6, 9, 12, and 18 months, and at early termination/study completion (up to 24 months)

Population: Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with at least one baseline and one post-baseline assessments.

'Total ADA Negative' is the sum of all subjects who tested negative for ADA in the 2 following categories: 'ADA Negative', those who are pre-dose ADA negative or are missing pre-dose ADA data and who have all negative post-dose ADA results; and 'ADA Negative (Treatment Unaffected)', a subset who are pre-dose ADA positive but do not have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement. 'Total ADA Positive' is the sum of all subjects who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Boosted)', those who are pre-dose ADA positive and have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement; and 'ADA Positive (Treatment Induced)', those who are pre-dose ADA negative or missing data and who have at least one post-dose ADA positive sample.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline Total ADA Negative (Neg + Neg Unaffected)	183 Participants
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline ADA Negative	180 Participants
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline ADA Negative (Treatment Unaffected)	3 Participants
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline Total ADA Positive (Boosted + Induced)	10 Participants
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline ADA Positive (Treatment Boosted)	2 Participants
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline ADA Positive (Treatment Induced)	8 Participants

SECONDARY outcome

Timeframe: Pre-dose at Weeks 2, 3, and 5, Months 3, 6, 12, and 18, and at treatment discontinuation (up to 2 years)

Population: Pharmacokinetic-Evaluable Population: all participants who received at least one dose of emicizumab and had at least one post-baseline emicizumab concentration result.

The trough concentration is a measure of the plasma concentration of a study drug at the end of of the dosage interval.

Outcome measures

Outcome measures
Measure	1.5 mg/kg Emicizumab QW n=193 Participants Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Week 2	16.7 micrograms per millilitre (μg/mL) Standard Deviation 5.5
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Week 5	52.4 micrograms per millilitre (μg/mL) Standard Deviation 15.5
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Month 3	52.5 micrograms per millilitre (μg/mL) Standard Deviation 18.3
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Month 6	53.4 micrograms per millilitre (μg/mL) Standard Deviation 19.3
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Month 12	54.2 micrograms per millilitre (μg/mL) Standard Deviation 20.6
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Month 18	53.6 micrograms per millilitre (μg/mL) Standard Deviation 18.3
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Week 3	30.7 micrograms per millilitre (μg/mL) Standard Deviation 9.2
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints Treatment Discontinuation	54.6 micrograms per millilitre (μg/mL) Standard Deviation 19.0

Adverse Events

1.5 mg/kg Emicizumab QW

Serious events: 31 serious events

Other events: 104 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	1.5 mg/kg Emicizumab QW n=193 participants at risk Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Blood and lymphatic system disorders THROMBOCYTOPENIA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Cardiac disorders ACUTE MYOCARDIAL INFARCTION	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Gastrointestinal disorders ABDOMINAL COMPARTMENT SYNDROME	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Gastrointestinal disorders DIVERTICULUM INTESTINAL HAEMORRHAGIC	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Gastrointestinal disorders GASTROINTESTINAL HAEMORRHAGE	0.52% 1/193 • Number of events 5 • From Baseline until study completion (up to 2 years)
Gastrointestinal disorders RETROPERITONEAL HAEMATOMA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Hepatobiliary disorders SUBCAPSULAR HEPATIC HAEMATOMA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations ABSCESS	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations APPENDICITIS	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations CATHETER SITE ABSCESS	1.0% 2/193 • Number of events 2 • From Baseline until study completion (up to 2 years)
Infections and infestations DEVICE RELATED INFECTION	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations INFECTED SKIN ULCER	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations PNEUMONIA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Infections and infestations WOUND INFECTION	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications FEMUR FRACTURE	1.0% 2/193 • Number of events 2 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications FIBULA FRACTURE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications FRACTURED SACRUM	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications HEAD INJURY	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications HIP FRACTURE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications INJURY	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications JOINT INJURY	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications LOWER LIMB FRACTURE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications MULTIPLE INJURIES	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications POST PROCEDURAL HAEMORRHAGE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications TIBIA FRACTURE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications WOUND DEHISCENCE	1.0% 2/193 • Number of events 2 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications WOUND HAEMATOMA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Musculoskeletal and connective tissue disorders HAEMARTHROSIS	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Musculoskeletal and connective tissue disorders HAEMATOMA MUSCLE	1.0% 2/193 • Number of events 2 • From Baseline until study completion (up to 2 years)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) ADENOCARCINOMA OF COLON	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) BLADDER NEOPLASM	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Nervous system disorders GENERALISED TONIC-CLONIC SEIZURE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Nervous system disorders TENSION HEADACHE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Product Issues DEVICE DISLOCATION	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Renal and urinary disorders NEPHROTIC SYNDROME	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Renal and urinary disorders RENAL PAIN	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Renal and urinary disorders URETEROLITHIASIS	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Respiratory, thoracic and mediastinal disorders EPISTAXIS	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Respiratory, thoracic and mediastinal disorders PNEUMONIA ASPIRATION	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Surgical and medical procedures CENTRAL VENOUS CATHETER REMOVAL	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Vascular disorders HAEMATOMA	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)
Vascular disorders HAEMORRHAGE	0.52% 1/193 • Number of events 1 • From Baseline until study completion (up to 2 years)

Other adverse events

Other adverse events
Measure	1.5 mg/kg Emicizumab QW n=193 participants at risk Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
General disorders INJECTION SITE REACTION	11.4% 22/193 • Number of events 29 • From Baseline until study completion (up to 2 years)
General disorders PYREXIA	10.9% 21/193 • Number of events 26 • From Baseline until study completion (up to 2 years)
Infections and infestations INFLUENZA	8.3% 16/193 • Number of events 25 • From Baseline until study completion (up to 2 years)
Infections and infestations NASOPHARYNGITIS	15.5% 30/193 • Number of events 47 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications FALL	6.2% 12/193 • Number of events 16 • From Baseline until study completion (up to 2 years)
Injury, poisoning and procedural complications LIMB INJURY	5.2% 10/193 • Number of events 12 • From Baseline until study completion (up to 2 years)
Musculoskeletal and connective tissue disorders ARTHRALGIA	17.1% 33/193 • Number of events 52 • From Baseline until study completion (up to 2 years)
Nervous system disorders HEADACHE	15.0% 29/193 • Number of events 52 • From Baseline until study completion (up to 2 years)
Respiratory, thoracic and mediastinal disorders COUGH	6.2% 12/193 • Number of events 13 • From Baseline until study completion (up to 2 years)

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800-821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The study being conducted under this agreement is part of the overall study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the study, but only after the first publication or presentation that involves the overall study. The Sponsor may request that confidential information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER